Objective: To present highlights from the Patient-Focused Drug Development (PFDD) meetings held at FDA for Parkinson’s disease (PD) and Huntington’s disease (HD) to raise awareness of the importance of listening to the needs of those living with movement disorders.

Background: Traditional drug development has not systematically incorporated patients’ perspectives and preferences into the process. In order to address this need, FDA’s PFDD meetings have been organized to more systematically gather patients’ perspectives on their condition and available therapies. On September 22, 2015, the FDA hosted a public PFDD meeting focused on HD and PD to gain patient feedback. 

Methods: 100 participants attended the FDA’s PFDD meeting for PD in person and >160 joined the four hour PD segment of the meeting remotely. Two-thirds of the participants who attended in person had a diagnosis of PD. Fifty participants attended the HD PFDD meeting in person and 60 participants provided input through the live webcast. The FDA heard directly from participants with an emphasis on symptoms, existing treatment options, and the unmet need for new therapies. 

Results: Participant comments focused on disease symptoms and daily impacts that matter most to patients and patient perspectives on treatments. Gathering of public input was extended for two months past the meeting; all feedback from the meeting and extension period, were compiled to prepare a voice-of-the-patient report for HD and PD that are publically available. This information will be integrated into evaluation of improved outcome measures and assessment of risk/benefit in future trials.

Conclusions: Engaging patients in a public forum fosters a thorough understanding of the impact caused by the diverse symptoms of these diseases from the patient perspective. The FDA’s PFDD initiative promises to identify unmet needs and aid in the FDA’s decision-making as it establishes the context in which it considers the risks and benefits associated with future treatments for HD and PD. This aligns with the goals of Critical Path Institute’s two precompetitive consortia Critical Path for Parkinson’s (CPP) and Huntington’s Disease Regulatory Science Consortium (HD-RSC) aimed at accelerating development of effective treatments through robust collaboration centered on data sharing, patient input and regulatory science.  

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MDS Abstracts - https://www.mdsabstracts.org/abstract/the-fda-is-listening-integrating-the-voice-of-the-patient-in-drug-development-for-parkinsons-and-huntingtons-diseases/